NEW YORK (CBSNewYork) — The hefty price tag of a new muscular dystrophy drug is stirring up a lot of controversy.
The pharmaceutical company is delaying the release of the drug after Washington lawmakers got involved.
Dylan Miceli Nelson has a rare form of muscular dystrophy called ‘Duchenne’ found almost exclusively in boys.
The deadly genetic disorder weakens his muscles over time, but for the past ten years, the steroid deflazacort has helped.
“This is a drug that likely adds three years of walking to boys. So, in all likelihood Dylan went into a wheelchair three years later than he would have otherwise,” Dr. Carrie Miceli explained.
As CBS2’s Dr. Max Gomez explained Dylan’s parents are also researchers at UCLA studying Duchenne.
Until now, patients like Dylan had to find a way to get the drug from overseas.
The FDA has approved the drug for sale in the U.S. and last week Marathon Pharmaceuticals was about to start selling it — until the company said it would charge $89,000 a year compared to just $1,200 for the generic version abroad.
The price tag has outraged lawmakers on Capitol Hill who vowed to put pressure on Marathon.
“Companies can charge us any price they want and that is why we are paying by far the most for medicine,” U.S. Sen. Bernie Sanders (I-Vermont) said.
The government has no federal oversight when it comes to pricing, but Sanders has called for an investigation.
“We are going to be their worst nightmare,” Sanders said.
Marathon commented on the concern Wednesday.
“We hope to come to a conclusion that brings the benefit of this medicine to all patients who need it regardless of their ability to pay, and enables us to continue further research,” they said in a statement.
“It would be nice to be transparent about what all the costs were, but it’s not inconceivable that the costs will be much higher than it might be in other countries,” Dr. Miceli said.
Dr. Micel said she hopes to strike a balance so more patients have access to the drug.
Experts said it’s not easy to obtain a drug from overseas, and that until now, only about 10 percent of patients have been able to take it.